Sarepta Therapeutics Inc. CEO Douglas Ingram said he expects “ferocious” demand for gene therapy Elevidys (delandistrogene moxeparvovec), granted full approval by the U.S. FDA for Duchenne muscular dystrophy (DMD). Shares of the Cambridge, Mass.-based firm closed June 21 at $16.72, up $37.22, or about 30% on the news. Read More
The BioWorld Biopharmaceutical Index (BBI) saw an 8.15% increase at the close of May, outperforming the Dow Jones Industrial Average, which rose by 2.64%, and the Nasdaq Biotechnology Index, which was up by 0.7%. The BBI previously showed a strong performance in Q1, followed by a dip for all three indices in April, before all rebounded in May. Read More
Aprilbio Co. Ltd. saw its stock surge 30% June 20 after announcing a potential $475 million (₩655.88 billion) deal with Evommune Inc. for autoimmune disease drug candidate APB-R3. Read More
Communication missteps, overreliance on a contract research organization and lack of clarity of U.S. FDA expectations for an expanded access protocol for emergency use resulted in a warning letter posted June 18 that highlights potential pitfalls for sponsor/investigators conducting the individualized studies. Read More
Newco Yellowstone Biosciences Ltd. has been formed to develop soluble bispecific T-cell receptors against a novel class of tumor-specific antigens it has discovered in leukemia patients who were cured by a donor stem cell transplant. The company has proprietary access to a biobank of over 10,000 samples from more than 3,000 acute myeloid leukemia (AML) patients. A small number of these patients were cured by an allogeneic bone marrow transplant, whilst unusually, having no sign of graft-vs.-host disease. Read More
In May, the U.S. FDA approved 11 new drugs, a significant drop from the 26 approvals in April, which was the sixth-highest monthly total since 2016. This also falls short of March’s record-setting 30 approvals, the highest number recorded by BioWorld. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Aphaia, Atai, Cessation, Gilead, Janssen, Jazz, Kiromic, Mendus, PTC, Silence, Vaxxinity. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Avextra, Medigene, SAB, Synqube, Vanda. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Astrazeneca, Innocare, Neurotech, PTC. Read More
